A Phase 1/2 Study of NRTX-1001 Neuronal Cell Therapy in Drug-Resistant Bilateral Mesial Temporal Lobe Epilepsy (MTLE)
A Phase 1/2 Study of NRTX-1001 Neuronal Cell Therapy in Drug-Resistant Bilateral Temporal Lobe Epilepsy (MTLE)
Neurona Therapeutics
10 participants
Nov 14, 2024
INTERVENTIONAL
Conditions
Summary
This is a multicenter, single arm, open label clinical trial that is designed to test the safety and preliminary efficacy of single administration inhibitory nerve cells called interneurons (NRTX-1001), into both temporal lobes of subjects with drug-resistant bilateral mesial temporal lobe epilepsy.
Eligibility
Inclusion Criteria12
- Male or female, age 18-75 years.
- Subjects of childbearing potential will use highly effective contraception.
- Proven history of focal seizures of hippocampal origin with bilateral seizure foci confirmed by scalp or intracranial ictal EEG (including confirmation by recordings from responsive neurostimulation \[RNS\] electrodes when applicable).
- Either
- bilateral hippocampal sclerosis on MRI (evidenced by increased FLAIR signal intensity in both hippocampi or by visual assessment showing reduced volume compared to normal) or
- bilateral temporal hypometabolism on 18-Flourodeoxyglucose Positron Emission Tomography (FDG PET) (assessed by visual assessment, comparing temporal regions to frontal/parietal lobe neocortex). In this case, ictal EEG recordings must also include intracranial confirmation.
- or
- a combination of unilateral instances of the evidence described in a. and b. (e.g., one side can be evidenced by criterion a. and the other side by criterion b.) MRI or PET scans used for assessment must have been acquired within 3 years of screening.
- Subject has had at least four clinical focal seizures, including at least two clinical focal seizures with objective manifestations, on average, per month for the 6 months prior to screening.
- Subject has previously had adequate (in opinion of investigator) therapeutic trials of at least two Anti-Seizure Medicines (ASMs).
- Current ASM regimen, and doses of other drugs known to affect seizure frequency (e.g., antidepressants), have been stable for at least three months prior to enrollment.
- Subject can converse and read in English or Spanish. Able to participate in required study procedures and provide signed informed consent.
Exclusion Criteria11
- Epilepsy due to other and/or progressive neurologic disease.
- Evidence of seizure focus outside hippocampus (either by seizure semiology or EEG findings).
- MRI indicating potential malignant lesion (low-grade glioma of any type is excluded) in any location or non-malignant potentially epileptogenic lesion outside the hippocampus. Small (\<2 cm) non invasive meningioma, remote from the affected temporal lobe, is not exclusionary.
- Seizures of non-focal origin.
- History of status epilepticus in the year prior to screening, as guided by ILAE criteria (Trinka 2015) in the judgement of the PI. A history of cluster seizures is permitted.
- Psychogenic Non-Epileptic Seizures (PNES) within the past 3 years.
- Severe psychiatric disorders.
- Primary or secondary immunodeficiency.
- Pregnancy, or currently breastfeeding.
- Suicide attempts in past year.
- Significant other medical conditions which would impair safe participation.
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Interventions
Biological: NRTX-1001 is an inhibitory neural cell therapy investigational product. It is derived from a human stem cell line that has been converted into high-purity inhibitory interneurons that produce GABA. NRTX-1001 is intended to persist long-term and not require repeated administration.
Locations(17)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06422923